Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

Last updated: August 16, 2024
Sponsor: Alexion Pharmaceuticals, Inc.
Overall Status: Completed

Phase

3

Condition

Aplastic Anemia

Anemia

White Cell Disorders

Treatment

Ravulizumab

Ravulizumab OBDS

Clinical Study ID

NCT03748823
ALXN1210-PNH-303
2017-002370-39
  • Ages > 18
  • All Genders

Study Summary

The primary objective of this study is to evaluate pharmacokinetics (PK) of ravulizumab administered subcutaneously via an on-body delivery system (OBDS) compared with intravenously administered ravulizumab in adult participants with PNH who are clinically stable on eculizumab for at least 3 months prior to study entry.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Male or female ≥18 years of age

  • Treated with eculizumab for PNH for at least 3 months prior to Day 1

  • LDH level ≤1.5 × upper limit of normal (ULN) at screening

  • PNH diagnosis confirmed by documented high-sensitivity flow cytometry.

  • Documented meningococcal vaccination not more than 3 years prior to, or at the timeof, initiating study treatment.

  • Body weight ≥40 to <100 kilogram (kg)

  • Female participants of childbearing potential must use highly effectivecontraception starting at screening and continuing until at least 8 months after thelast dose of ravulizumab.

  • Willing and able to give written informed consent and comply with study visitschedule.

Exclusion

Exclusion Criteria:

  • More than 1 LDH value > 2 × ULN within the 3 months prior to study entry

  • History of bone marrow transplantation.

  • History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic diseasethat, in the opinion of the Investigator or Sponsor, would preclude participation.

  • Unstable medical conditions (for example, myocardial ischemia, activegastrointestinal bleed, severe congestive heart failure, anticipated need for majorsurgery within 6 months of randomization, coexisting chronic anemia unrelated toPNH).

  • Females who are pregnant, breastfeeding or who have a positive pregnancy test atscreening or Day 1.

  • Participation in another interventional clinical study or use of any experimentaltherapy within 30 days before initiation of study drug on Day 1 in this study orwithin 5 half-lives of that investigational product, whichever is greater.

Study Design

Total Participants: 139
Treatment Group(s): 2
Primary Treatment: Ravulizumab
Phase: 3
Study Start date:
February 19, 2019
Estimated Completion Date:
August 31, 2023

Study Description

The study will consist of up to a 30-day Screening Period, a 10-week Randomized Treatment Period, and an Extension Period of up to 172 weeks.

Connect with a study center

  • Clinical Trial Site

    Liverpool,
    Australia

    Site Not Available

  • Research Site

    Liverpool, 2170
    Australia

    Site Not Available

  • Clinical Trial Site

    Parkville,
    Australia

    Site Not Available

  • Research Site

    Parkville, 3050
    Australia

    Site Not Available

  • Clinical Trial Site

    Vienna,
    Austria

    Site Not Available

  • Research Site

    Vienna, 1090
    Austria

    Site Not Available

  • Clinical Trial Site

    Antwerpen,
    Belgium

    Site Not Available

  • Research Site

    Antwerpen, 2020
    Belgium

    Site Not Available

  • Clinical Trial Site

    Brussels,
    Belgium

    Site Not Available

  • Research Site

    Bruxelles, 1200
    Belgium

    Site Not Available

  • Clinical Trial Site

    Hasselt,
    Belgium

    Site Not Available

  • Research Site

    Hasselt, 3500
    Belgium

    Site Not Available

  • Clinical Trial Site

    Leuven,
    Belgium

    Site Not Available

  • Research Site

    Leuven, 3000
    Belgium

    Site Not Available

  • Clinical Trial Site

    Botucatu,
    Brazil

    Site Not Available

  • Research Site

    Botucatu, 18618-970
    Brazil

    Site Not Available

  • Clinical Trial Site

    Ribeirão Preto,
    Brazil

    Site Not Available

  • Research Site

    Ribeirão Preto, 14049-901
    Brazil

    Site Not Available

  • Research Site

    Rio De De Janeiro, 20211030
    Brazil

    Site Not Available

  • Clinical Trial Site

    Rio De Janeiro,
    Brazil

    Site Not Available

  • Clinical Trial Site

    Salvador,
    Brazil

    Site Not Available

  • Research Site

    Salvador, 40170010
    Brazil

    Site Not Available

  • Research Site

    Sao Paulo, 05403-000
    Brazil

    Site Not Available

  • Clinical Trial Site

    São Paulo,
    Brazil

    Site Not Available

  • Research Site

    São Paulo, 01308-050
    Brazil

    Site Not Available

  • Research Site

    Toronto, Ontario M5G 2C4
    Canada

    Site Not Available

  • Clinical Trial Site

    Toronto,
    Canada

    Site Not Available

  • Clinical Trial Site

    Helsinki,
    Finland

    Site Not Available

  • Research Site

    Helsinki, 00029
    Finland

    Site Not Available

  • Clinical Trial Site

    Amiens,
    France

    Site Not Available

  • Research Site

    Amiens, 80054
    France

    Site Not Available

  • Clinical Trial Site

    Brest,
    France

    Site Not Available

  • Research Site

    Brest, 29609
    France

    Site Not Available

  • Clinical Trial Site

    Lille,
    France

    Site Not Available

  • Research Site

    Lille, 59037
    France

    Site Not Available

  • Clinical Trial Site

    Montpellier,
    France

    Site Not Available

  • Research Site

    Montpellier Cedex 5, 34090
    France

    Site Not Available

  • Clinical Trial Site

    Nantes,
    France

    Site Not Available

  • Research Site

    Nantes cedex 01, 44093
    France

    Site Not Available

  • Clinical Trial Site

    Nice,
    France

    Site Not Available

  • Research Site

    Nice, 6200
    France

    Site Not Available

  • Clinical Trial Site

    Paris,
    France

    Site Not Available

  • Research Site

    Paris, 75010
    France

    Site Not Available

  • Clinical Trial Site

    Pessac,
    France

    Site Not Available

  • Research Site

    Pessac, F-33604
    France

    Site Not Available

  • Research Site

    Pierre Benite Cedex, 69495
    France

    Site Not Available

  • Clinical Trial Site

    Pierre-Bénite,
    France

    Site Not Available

  • Clinical Trial Site

    Poitiers,
    France

    Site Not Available

  • Research Site

    Poitiers, 86021
    France

    Site Not Available

  • Clinical Trial Site

    Rennes,
    France

    Site Not Available

  • Research Site

    Rennes Cedex 9, 35033
    France

    Site Not Available

  • Clinical Trial Site

    Strasbourg,
    France

    Site Not Available

  • Research Site

    Strasbourg, 67098
    France

    Site Not Available

  • Clinical Trial Site

    Tours,
    France

    Site Not Available

  • Research Site

    Tours, 37044
    France

    Site Not Available

  • Clinical Trial Site

    Catania,
    Italy

    Site Not Available

  • Research Site

    Catania, 95123
    Italy

    Site Not Available

  • Clinical Trial Site

    Milano,
    Italy

    Site Not Available

  • Research Site

    Milano, 20122
    Italy

    Site Not Available

  • Clinical Trial Site

    Roma,
    Italy

    Site Not Available

  • Research Site

    Roma, 00168
    Italy

    Site Not Available

  • Research Site

    Rome, 161
    Italy

    Site Not Available

  • Clinical Trial Site

    Maastricht,
    Netherlands

    Site Not Available

  • Research Site

    Maastricht, 6229 HX
    Netherlands

    Site Not Available

  • Clinical Trial Site

    Ekaterinburg,
    Russian Federation

    Site Not Available

  • Research Site

    Ekaterinburg, 620102
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Moscow,
    Russian Federation

    Site Not Available

  • Research Site

    Moscow, 125284
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Saint Petersburg,
    Russian Federation

    Site Not Available

  • Research Site

    Saint-Petersburg, 197089
    Russian Federation

    Site Not Available

  • Research Site

    Badalona, 8916
    Spain

    Site Not Available

  • Clinical Trial Site

    Barcelona,
    Spain

    Site Not Available

  • Research Site

    Barcelona, 08036
    Spain

    Site Not Available

  • Clinical Trial Site

    Donostia,
    Spain

    Site Not Available

  • Research Site

    Donostia, 20014
    Spain

    Site Not Available

  • Clinical Trial Site

    Las Palmas De Gran Canaria,
    Spain

    Site Not Available

  • Research Site

    Las Palmas de Gran Canaria, 35020
    Spain

    Site Not Available

  • Clinical Trial Site

    Madrid,
    Spain

    Site Not Available

  • Research Site

    Madrid, 28040
    Spain

    Site Not Available

  • Clinical Trial Site

    Majadahonda,
    Spain

    Site Not Available

  • Research Site

    Majadahonda, 28222
    Spain

    Site Not Available

  • Clinical Trial Site

    Sevilla,
    Spain

    Site Not Available

  • Research Site

    Sevilla, 41013
    Spain

    Site Not Available

  • Clinical Trial Site

    Uppsala,
    Sweden

    Site Not Available

  • Research Site

    Uppsala, 75185
    Sweden

    Site Not Available

  • Clinical Trial Site

    Adana,
    Turkey

    Site Not Available

  • Research Site

    Adana, 01330
    Turkey

    Site Not Available

  • Research Site

    Istambul, 34899
    Turkey

    Site Not Available

  • Clinical Trial Site

    Istanbul,
    Turkey

    Site Not Available

  • Research Site

    Istanbul, 34096
    Turkey

    Site Not Available

  • Research Site

    Izmir, 35100
    Turkey

    Site Not Available

  • Research Site

    İstanbul, 34093
    Turkey

    Site Not Available

  • Clinical Trial Site

    İzmir,
    Turkey

    Site Not Available

  • Clinical Trial Site

    Los Angeles, California 90033
    United States

    Site Not Available

  • Research Site

    Los Angeles, California 90089
    United States

    Site Not Available

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